Barth syndrome is a rare genetic disease in boys that can cause life-threatening heart failure and also weakens the skeletal muscles and the immune system. There is no specific treatment, but new research, involving new mouse models, shows the potential of a gene therapy approach in preventing and reversing cardiac dysfunction in Barth syndrome.
from Health & Medicine News -- ScienceDaily https://www.sciencedaily.com/releases/2020/03/200309165231.htm
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